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Spark Therapeutics, Inc. (ONCE) RSS Feed

Followers
12
Posters
27
Posts (Today)
0
Posts (Total)
124
Created
01/30/15
Type
Free
Moderators
Spark Therapeutics, Inc. is engaged in the field of gene therapy. The Company develops one-time, life-altering treatments for patients suffering from debilitating genetic diseases. The Company is focused on treating rare diseases where no, or only palliative therapies exist. The Company addresses a range of debilitating genetic diseases including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Its pipeline of product candidates is based on AAV vector technology. The Company’s product candidate, SPK-RPE65, is in a Phase III clinical trial for the treatment of inherited retinal dystrophies (IRDs) caused by mutations in the RPE65 gene. It is also conducting other preclinical programs such as SPK-CHM, SPK-FIX and GenableRhoNova. The Company has received both breakthrough therapy and orphan product designation for SPK-RPE65 and orphan product designation for SPK-CHM for the treatment of choroideremia. http://www.sparktx.com
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